Cystic Fibrosis (CF) is a single gene, recessive disorder characterized by a defective cAMP stimulated chloride conductance across epithelia surfaces, especially in the lung and pancreatic duct. Clinically, this defect results in decreased mucocilliary clearance in lung airways, leading to chronic bacterial infections and inflammation. As a result, patients have a life expectancy of less than 30 years. Clinical trials have thus far focused on either gene or drug therapies for the pulmonary treatment of cystic fibrosis [7,8].
The most common CF mutation, .DELTA.F508, results in failure of the CFTR protein to reach the plasma membrane, likely due to protein trafficking error. The action of 4PBA has been shown to restore CFTR chloride conductance on the plasma membrane of .DELTA.F508 bronchial epithelial cells in vitro [10].